• Respiratory Medicine
• Paediatric Emergency Medicine
• Pathology
• Therapeutics

The stupendous scientific advances in understanding disease in children have delivered really spectacular treatment advances, highly effective modulator therapies (HEMTs) in cystic fibrosis (CF) being an obvious example. But clearly, the essential pre-requisite for this sort of precision medicine is a thorough understanding of the biology of the disease. Ivacaftor was the first specific molecular therapy for CF but it is only effective as a single agent in class III mutations. Were it to have been given to all children with a chronic productive cough, or even all children with CF, it would likely have been discarded as inactive. Asthma treatments of proven value (inhaled and oral corticosteroids, and the monoclonal mepolizumab, which binds to circulating interleukin 5 (IL-5) and thus reduces airway eosinophilic inflammation) were in danger of being discarded as inactive because they were initially deployed uncritically to all comers.1 If therapeutic progress is to be made, we must look beyond umbrella, descriptive terms like ‘asthma’ by asking ‘what sort of asthma does this child have’, an approach which has led to the successful deployment of omalizumab, mepolizumab and dupilumab in severe asthma. However, we do need to move to better understanding of endotypes and move away from sterile N-of-1 treatment trials to focused, targeted therapies, inspired by the use of HEMT in CF clinics.

Sadly, many paediatricians have historically seemed reluctant to engage with basic scientists to obtain the evidence to inform real precision medicine in other respiratory diseases. Acute attacks of breathlessness across the developmental spectrum are a case in point. We …